PubMed Journals Articles About "Therachon Receives Orphan Drug Designation Achondroplasia" RSS

08:00 EDT 24th March 2018 | BioPortfolio

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Showing "Therachon receives orphan drug designation achondroplasia" PubMed Articles 1–25 of 9,200+

Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe.

In the European Union demonstration of 'significant benefit' is mandatory if satisfactory methods exist for a disease targeted by a new orphan medicinal product. Significant benefit is required at the time of orphan designation, when it can be supported by preclinical studies, and at the time of marketing authorization, when clinical data are needed. For the first time, our work has identified, defined and organized the scientific grounds on which significant benefit is granted in the European Union, based ...

The Use of Social Media in Orphan Drug Development.

Social media has transformed how people interact with one another through the Internet, and it has the potential to do the same for orphan drug development. Currently, social media influences the orphan drug development process in the following three ways: assisting the study of orphan diseases, increasing the awareness of orphan disease, and playing a vital role in clinical trials. However, there are some caveats to the utilization of social media, such as the need to protect patient privacy by adequately ...

Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology.

Developing drugs for rare disease can be challenging due to specific rare disease characteristics. The French Medical Pharmacology is structured and positioned to play a major role in Orphan Drug Research and Development due to the required expertise concentrated into pharmacology departments, exclusively implemented within the French University Hospitals, public hospitals that are linked to a medical school (and often a pharmacy school) with numerous INSERM or CNRS labelled research units. In addition, the...

Growth velocity and biological variables during puberty in achondroplasia.

Achondroplasia is the most common form of inherited disproportionate short stature. Cross-sectional design studies of height show that, during childhood, height standard deviation scores (SDS) declines steadily and reaches a mean adult height at -6.42 and -6.72 SDS. However, there is a lack of knowledge about longitudinal growth and biological variables during puberty for children with achondroplasia. Here we report the growth velocity and biological parameters during puberty in children with achondroplasia...

Sinus pericranii in achondroplasia: a case report and review of the literature.

In the field of dysmorphology, achondroplasia is a well-known disorder. Sinus pericranii (SP), however, is not. The latter condition is a rare vascular malformation characterized by abnormal connections between the intracranial and the extracranial venous drainage pathways. The etiology of SP remains unclear, and yet, these defects can be present at birth, develop spontaneously later, or evolve following head trauma. Here, we report on a 2-year-old male with achondroplasia, SP, and craniocervical junction s...

Humeral lengthening in patients with achondroplasia and in patients with post-septic shortening: comparison of procedure efficiency and safety.

Although humeral lengthening in patients with achondroplasia is an accepted procedure for improving functional status, there is still a paucity of information about the effectiveness of the method. Therefore, the aim of this study was to evaluate the efficacy and safety of humeral lengthening using monolateral fixators in patients with achondroplasia and unilateral shortening.

Diabetes Drug Receives Boxed Warning for Increased Risk of Leg and Foot Amputations.

Progesterone for Prevention of Preterm Birth: Shortcomings and Unintended Consequences of the Orphan Drug Act.

Preterm birth is a problem of major public health significance that continues to plague our country despite the existence of a therapy, 17α-hydroxyprogesterone caproate, with known efficacy in reducing the risk of spontaneous preterm birth among high-risk women. Over the past several years, the Louisiana Department of Health has undertaken a robust, multifaceted initiative to improve access to 17α-hydroxyprogesterone caproate, which resulted in a 3.5-fold increase in the percentage of eligible high-risk p...

Cutaneous T-cell lymphomas: Focusing on novel agents in relapsed and refractory disease.

Patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL) display a dismal prognosis and their therapy represents an unmet medical need, as the best treatment strategy is yet to be determined. Exciting data on novel targeted agents are now emerging from recently concluded and ongoing clinical trials in patients with relapsed and refractory CTCL. Three FDA approved compounds are used as single agents including the oral retinoid bexarotene and histone deacetylase inhibitors romidepsin and vorinost...

Optimal non-invasive diagnosis of fetal achondroplasia combining ultrasonography and circulating cell-free fetal DNA analysis.

To assess the performance of non-invasive prenatal testing of achondroplasia using high-resolution melting (HRM) analysis. To propose an optimal diagnosis strategy combining ultrasound scan and cell-free fetal DNA (cffDNA) analysis.

Loss of Magnet® Designation and Changes in RN Work Engagement: A Report on How 1 Hospital's Culture Changed Over Time.

The aim of this study was to examine the loss of Magnet® designation and how RNs' work engagement changed at 1 community hospital.

Orphan G protein-coupled receptors: The role in CNS disorders.

There are various types of receptors in the central nervous system (CNS). G protein-coupled receptors (GPCRs) have the highest expression with a wide range of physiological functions. A newer sub group of these receptors namely orphan GPCRs have been discovered. GPR3, GPR6, GPR17, GPR26, GPR37, GPR39, GPR40, GPR50, GPR52, GPR54, GPR55, GPR85, GPR88, GPR103, and GPR139 are the selected orphan GPCRs for this article. Their roles in the central nervous system have not been understood well so far. However, rece...

Reolysin and Histone Deacetylase Inhibition in the Treatment of Head and Neck Squamous Cell Carcinoma.

Oncolytic viruses (OVs) are emerging as powerful anti-cancer agents and are currently being tested for their safety and efficacy in patients. Reovirus (Reolysin), a naturally occurring non-pathogenic, double-stranded RNA virus, has natural oncolytic activity and is being tested in phase I-III clinical trials in a variety of tumor types. With its recent US Food and Drug Administration (FDA) orphan drug designation for several tumor types, Reolysin is a potential therapeutic agent for various cancers, includi...

Nuclear orphan receptor NR2F6 as a safeguard against experimental murine colitis.

Nuclear receptors are known to regulate both immune and barrier functions in the GI tract. The nuclear orphan receptor NR2F6 has been shown to suppress the expression of proinflammatory cytokines in T lymphocytes. NR2F6 gene expression is reduced in patients with IBS or UC, but its functional role and tissue dependency in healthy and inflamed gut have not yet been investigated.

A systems-level analysis of drug-target-disease associations for drug repositioning.

Drug repositioning is the process of finding new therapeutic uses for existing, approved drugs-a process thathas value when considering the exorbitant costs of novel drug development. Several computational strategies exist as a way to predict these alternative applications. In this study, we used datasets on: (1) human biological drug targets and (2) disease-associated genes and, based on a direct functional interaction between them, searched for potential opportunities for drug repositioning. From the set ...

So you want to be a Fellow in the American Association of Nurse Practitioners: Navigating the Fellowship process to ensure your success.

The professional designation as Fellow of the American Association of Nurse Practitioners (FAANP) is the highest professional designation for any nurse practitioner (NP). The Fellows of the American Association of Nurse Practitioners (AANP) operate the FAANP program and annually invite colleagues that have made a significant impact on the NP profession for consideration of this prestigious designation. Since its inception in 2000, nearly 700 NPs, or less than 1% of the membership, have been inducted as FAAN...

Perioperative Anaphylaxia on Drugs.

The diagnostic evaluation of perioperative anaphylaxia is of great importance for the medical care of the affected persons in the context of renewed interventions in general anesthesia. The most frequent triggers include muscle relaxants, antibiotics, latex and analgesics of the type of the cyclooxygenase inhibitors, but other perioperatively administered drugs such as opioids, anesthetics, benzodiazepines, dyes, disinfectants and even corticosteroids or drug additives may also be the cause in rare cases. T...

A comparison of the pharmacokinetic and pharmacodynamic properties of nitroglycerin according to the composition of the administration set: A preliminary study.

There is a risk of drug sorption into an intravenous administration set composed of polyvinyl chloride (PVC), polyurethane (PU), or polyolefin (PO). This has implications on the dose of the active ingredient the patient receives, and thus therapeutic success. This study aimed to determine the plasma concentration of nitroglycerin and the effect of nitroglycerin on patients based on the composition of the administration set.

Proposed Industry Best Practices in Development and Marketing of Medical Foods for the Management of Chronic Conditions and Diseases while Awaiting Regulation.

Ideal therapeutics have low toxicity and can effectively manage condition(s) or disease(s). The Food & Drug Administration (FDA) marketing category of therapeutics called “medical foods” (MFs) meets such a definition. Medical foods have existed in Federal law since passage the Orphan Drug Act in 1988, which created a category of nutritional therapeutics separate from drugs. Unfortunately, MFs are not widely understood by the medical community or utilized in all patients who need them due to lack of a FD...

Use of coercion at Danish psychiatric wards by day of week and time of day.

Mechanical restraint and other forms of coercion may be used under certain circumstances at psych-iatric wards. The use of coercion receives considerable focus, yet little is known about its diurnal distribution. We examined the initiation of mechanical restraint, sedative drug administration, physical retention, door locking and personal shielding by time of day and week.

Preferences of Patients and Pharmacists with Regard to the Management of Drug-Drug Interactions: A Choice-Based Conjoint Analysis.

The management of drug-drug interactions (DDIs) is a complex process in which risk-benefit assessments should be combined with the patient's perspective.

BRIGHT brushing initiative receives £1.9 million.

Imaging of Skeletal Disorders Caused by Fibroblast Growth Factor Receptor Gene Mutations.

Fibroblast growth factors and fibroblast growth factor receptors (FGFRs) play important roles in human axial and craniofacial skeletal development. FGFR1, FGFR2, and FGFR3 are crucial for both chondrogenesis and osteogenesis. Mutations in the genes encoding FGFRs, types 1-3, are responsible for various skeletal dysplasias and craniosynostosis syndromes. Many of these disorders are relatively common in the pediatric population, and diagnosis is often challenging. These skeletal disorders can be classified ba...

Preventable adverse drug events in critically ill HIV patients: Is the detection of potential drug-drug interactions a useful tool?

The aim of this study was to develop a strategy to identify adverse drug events associated with drug-drug interactions by analyzing the prescriptions of critically ill patients.

Regulation of ciliary function by fibroblast growth factor signaling identifies FGFR3-related disorders achondroplasia and thanatophoric dysplasia as ciliopathies.

Cilia project from almost every cell integrating extracellular cues with signaling pathways. Constitutive activation of FGFR3 signaling produces the skeletal disorders achondroplasia (ACH) and thanatophoric dysplasia (TD), but many of the molecular mechanisms underlying these phenotypes remain unresolved. Here, we report in vivo evidence for significantly shortened primary cilia in ACH and TD cartilage growth plates. Using in vivo and in vitro methodologies, our data demonstrate that transient versus sustai...

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